Palovarotene in fibrodysplasia ossificans progressiva: review and perspective.

IF 2.5 3区医学 Q3 PHARMACOLOGY & PHARMACY

Expert Opinion on Pharmacotherapy Pub Date : 2025-01-20 DOI:10.1080/14656566.2025.2452938

Vincent A Verheij, Robert J Diecidue, Esmée Botman, Joseph D Harrington, Nobuhiko Haga, Alberto Hidalgo-Bravo, Patricia L R Delai, Vrisha Madhuri, Mona Al Mukaddam, Keqin Zhang, Tae-Joon Cho, Rolf Morhart, Richard Keen, Carmen L De Cunto, Clive S Friedman, Zvi Grunwald, Michael Zasloff, J Coen Netelenbos, Edward Hsiao, Frederick S Kaplan, Robert J Pignolo, Christiaan Scott, Elisabeth Marelise W Eekhoff

{"title":"Palovarotene in fibrodysplasia ossificans progressiva: review and perspective.","authors":"Vincent A Verheij, Robert J Diecidue, Esmée Botman, Joseph D Harrington, Nobuhiko Haga, Alberto Hidalgo-Bravo, Patricia L R Delai, Vrisha Madhuri, Mona Al Mukaddam, Keqin Zhang, Tae-Joon Cho, Rolf Morhart, Richard Keen, Carmen L De Cunto, Clive S Friedman, Zvi Grunwald, Michael Zasloff, J Coen Netelenbos, Edward Hsiao, Frederick S Kaplan, Robert J Pignolo, Christiaan Scott, Elisabeth Marelise W Eekhoff","doi":"10.1080/14656566.2025.2452938","DOIUrl":null,"url":null,"abstract":"Introduction: Palovarotene is a retinoic acid receptor gamma agonist that was studied in phase-2 and phase-3 clinical trials for the inhibition of new heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP). Despite numerous setbacks and regulatory delays, palovarotene is now the first approved FOP treatment in the U.S.A. Canada and Australia but remains unapproved in Europe where concerns surrounding the drug and its path to regional market authorization persist.Areas covered: The developmental history of palovarotene and an overview of the clinical trials and the regulatory approval journey are discussed by global FOP experts.Expert opinion: While post hoc analyses indicate that palovarotene may have modest benefits for the inhibition of new HO formation in FOP, a number of limitations and concerns remain about its generalized use. Although the long-term risks and benefits of treatment with palovarotene remain unknown, the regional approval of palovarotene marks a milestone for the FOP community at the very beginning of a new era of clinical trials.","PeriodicalId":12184,"journal":{"name":"Expert Opinion on Pharmacotherapy","volume":" ","pages":""},"PeriodicalIF":2.5000,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Opinion on Pharmacotherapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/14656566.2025.2452938","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}

引用次数: 0

Abstract

Introduction: Palovarotene is a retinoic acid receptor gamma agonist that was studied in phase-2 and phase-3 clinical trials for the inhibition of new heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP). Despite numerous setbacks and regulatory delays, palovarotene is now the first approved FOP treatment in the U.S.A. Canada and Australia but remains unapproved in Europe where concerns surrounding the drug and its path to regional market authorization persist.

Areas covered: The developmental history of palovarotene and an overview of the clinical trials and the regulatory approval journey are discussed by global FOP experts.

Expert opinion: While post hoc analyses indicate that palovarotene may have modest benefits for the inhibition of new HO formation in FOP, a number of limitations and concerns remain about its generalized use. Although the long-term risks and benefits of treatment with palovarotene remain unknown, the regional approval of palovarotene marks a milestone for the FOP community at the very beginning of a new era of clinical trials.

查看原文本刊更多论文

帕罗罗汀在进行性骨化性纤维发育不良中的作用：综述与展望。

简介：Palovarotene是一种维甲酸受体γ激动剂，在2期和3期临床试验中被研究用于抑制进行性骨化纤维发育不良（FOP）的新异位骨化（HO）。尽管经历了许多挫折和监管延误，palovarotene现在是美国、加拿大和澳大利亚首个获批的FOP治疗药物，但在欧洲仍未获批，欧洲对该药物及其进入区域市场授权的途径仍然存在担忧。涵盖领域：由全球FOP专家讨论帕洛瓦罗汀的发展历史、临床试验概述和监管批准之旅。专家意见：虽然事后分析表明，palovarotene可能对抑制FOP中新HO的形成有一定的益处，但其广泛使用仍然存在一些限制和担忧。尽管palovarotene治疗的长期风险和益处仍然未知，但palovarotene的地区批准标志着FOP社区在临床试验新时代的开端具有里程碑意义。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Expert Opinion on Pharmacotherapy 医学-药学

CiteScore

5.60

自引率

3.10%

发文量

163

审稿时长

4-8 weeks

期刊介绍： Expert Opinion on Pharmacotherapy is a MEDLINE-indexed, peer-reviewed, international journal publishing review articles and original papers on newly approved/near to launch compounds mainly of chemical/synthetic origin, providing expert opinion on the likely impact of these new agents on existing pharmacotherapy of specific diseases.